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Amyotrophic Lateral SclerosisPhase 3NCT03127267

Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

A Phase 3 study testing Masitinib (6.0), Riluzole and Masitinib (4.5) in people with Amyotrophic Lateral Sclerosis, run by AB Science at 56 sites in 17 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS). Read more about this study

Who is it for?
Ages 18 to 81 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Masitinib (6.0), Riluzole and Masitinib (4.5)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
495 participants
Where
56 sitesin 17 countries
Study dates
2 Feb 2021to Dec 2027
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 81 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Main inclusion criteria include:

  • Patients diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria
  • Patient with a familial or sporadic ALS
  • ALS disease duration from diagnosis no longer than 24 months at the screening visit
  • Patient treated with a stable dose of riluzole (100 mg/day) for at least 12 weeks days prior to the baseline visit
  • Patient with an ALSFRS-R score progression between onset of the disease and screening of > 0.3 per month, confirmed with an ALSFRS-R score progression of ≥ 1 point during a 12-week run-in period between screening and randomization.
  • Patient with a score, at screening, of at least 26 overall, including a score of at least 3 on item #3 and at least 2 on each of the 12 ALSFRS-R individual component items and with a score, at randomization, of at least 2 on each of the 12 ALSFRS-R individual component items

Main exclusion criteria include:

  • Patient with dementia or significant neurological, psychiatric, systemic or organic disease, uncontrolled or that may interfere with the conduct of the trial or its results
  • Patient with a FVC < 60% predicted normal value for gender, height, and age at screening and baseline
  • Pregnant, or nursing female patient

Trial locations and contacts

56 sites in 17 countries. Contact the study team or a site near you to ask about taking part.

Clinical Study Coordinator contact

+33(0)147200014

clinical@ab-science.com

56 sites in 17 countries

France11 sites, 11 recruiting

  • CHU de Angers

    Angers

    Recruiting

  • Groupe Hospitalier Pellegrin Tripode

    Bordeaux

    Recruiting

  • Hôpital neurologique Pierre Wertheimer

    Bron

    Recruiting

  • CHU Gabriel Montpied

    Clermont-Ferrand

    Recruiting

  • CHU de Lille - Hopital Roger Salengro

    Lille

    Recruiting

  • CHU de Limoges - Hôpital Dupuytren

    Limoges

    Recruiting

  • CHU de Marseille - Hôpital de la Timone

    Marseille

    Recruiting

  • CHRU de Montpellier - Gui de Chauliac

    Montpellier

    Recruiting

  • CHU de Nancy - Hopital Central

    Nancy

    Recruiting

  • CHU Hôpital Pasteur Nice

    Nice

    Recruiting

  • CHRU de Tours - Hopital Bretonneau

    Tours

    Recruiting

Other trials in France

Italy11 sites, 11 recruiting

  • Ospedale Civile Sant'Agostino - Estense

    Baggiovara, Modena

    Recruiting

  • ASST degli Spedali Civili di Brescia

    Brescia

    Recruiting

  • Centro Clinico NeMO Fondazione Serena Onlus

    Gussago

    Recruiting

  • Clinico Nemo Center (Centro Clinico NeMO Milano)

    Milan

    Recruiting

  • IRCCS Istituto Auxologico Italiano

    Milan

    Recruiting

  • Istituti Clinici Scientifici Maugeri IRCCS

    Milan

    Recruiting

  • San Raffaele Hospital (Ospedale San Raffaele)

    Milan

    Recruiting

  • University Hospital Maggiore della Carita

    Novara

    Recruiting

  • Azienda Ospedale-Università Padova

    Padova

    Recruiting

  • IRCCS Mondino Foundation

    Pavia

    Recruiting

  • University Hospital in Turin (Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino)

    Torino

    Recruiting

Other trials in Italy

United States6 sites, 6 recruiting

  • Johns Hopkins Medicine Brain Science Institute

    Baltimore, Maryland, 21205

    Recruiting

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

    Recruiting

  • Lahey Hospital and Medical Center

    Burlington, Massachusetts, 01805

    Recruiting

  • University of Virginia Health System

    Charlottesville, Virginia, 22903

    Recruiting

  • University of Kentucky

    Lexington, Kentucky, 40506

    Recruiting

  • University of Southern California

    Los Angeles, California, 90007

    Recruiting

Other trials in United States

Spain6 sites, 6 recruiting

  • Hospital General Universitario de Alicante

    Alicante

    Recruiting

  • Hospital Universitari de Bellvitge

    Barcelona

    Recruiting

  • Hospital Carlos III

    Madrid

    Recruiting

  • Hospital San Rafael

    Madrid

    Recruiting

  • Clinical Hospital Santiago de Compostela

    Santiago de Compostela

    Recruiting

  • Hospital Universitario y Politecnico La Fe

    Valencia

    Recruiting

Other trials in Spain

Greece4 sites, 4 recruiting

  • Athens Naval Hospital

    Athens

    Recruiting

  • Eginition Hospital

    Athens

    Recruiting

  • University General Hospital of Larissa

    Larissa

    Recruiting

  • General University Hospital of Patras

    Rio

    Recruiting

Other trials in Greece

Sweden3 sites, 3 recruiting

  • Centralsjukhuset Karlstad (Central Hospital Karlstad)

    Karlstad

    Recruiting

  • Skåne University Hospital

    Malmö

    Recruiting

  • Norrlands universitetssjukhus

    Umeå

    Recruiting

Other trials in Sweden

Ukraine3 sites, 3 recruiting

  • The State Institution of Neurology, Psychiatry and Narcology of NAMS of Ukraine

    Kharkiv

    Recruiting

  • Medical Center of LLC Medical Center Dopomoga Plus

    Kyiv

    Recruiting

  • Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurological Department

    Lviv

    Recruiting

Other trials in Ukraine

Israel2 sites, 2 recruiting

  • Hadassah University Hospital

    Jerusalem

    Recruiting

  • Tel-Aviv Medical Center Hôpital Sourasky (ICHILOV)

    Tel Aviv

    Recruiting

Other trials in Israel

Russia2 sites, 2 recruiting

  • Moscow city clinical Hospital after V.M. Buyanov

    Moscow

    Recruiting

  • Scientific Practical Medical Center "Innovation and Health"

    Novosibirsk

    Recruiting

Other trials in Russia

Belgium1 site, 1 recruiting

  • University Hospital Leuven (UZ Leuven)

    Leuven

    Recruiting

Other trials in Belgium

Denmark1 site, 1 recruiting

  • Bispebjerg Hospital

    Copenhagen

    Recruiting

Other trials in Denmark

Germany1 site, 1 recruiting

  • Department of Neurology, University of Ulm

    Ulm, 89081

    Not yet recruiting

Other trials in Germany

Norway1 site, 1 recruiting

  • Oslo University Hospital HF Ullevål

    Oslo

    Recruiting

Other trials in Norway

Poland1 site, 1 recruiting

  • Centrum Medyczne Neuromed

    Bydgoszcz

    Recruiting

Other trials in Poland

Portugal1 site, 1 recruiting

  • Hospital de Santa Maria

    Lisbon

    Recruiting

Other trials in Portugal

Serbia1 site, 1 recruiting

  • Clinical Centre of Serbia

    Belgrade

    Recruiting

Other trials in Serbia

Slovenia1 site, 1 recruiting

  • Klinicni center Ljubljana

    Ljubljana

    Recruiting

Other trials in Slovenia

About this study

Official title: Phase 3 Study to Compare the Efficacy and Safety of Masitinib in Combination With Riluzole Versus Placebo in Combination With Riluzole in the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)

The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Read the detailed description

Masitinib is a selective, oral tyrosine kinase inhibitor with neuroprotective capability demonstrated via numerous preclinical studies. Two of masitinib's main cellular targets are the mast cell and microglia cell. It is well-established that mast cells play a prominent role in neuroinflammatory processes. Microglia, resident immune cells of the central nervous system (CNS), also constitute an important source of neuroinflammatory mediators and may have fundamental roles in numerous neurodegenerative disorders. The development of masitinib in ALS is therefore based on the pharmacological action of masitinib in microglia cells and mast cells, thereby slowing microglial-related disease progression, reducing neuro-inflammation, and modulating the neuronal microenvironment in both central and peripheral nervous systems. This is a multicenter, double-blind, randomized, placebo-controlled, parallel-group (two ascending dose titrations of masitinib and matching placebo), comparative study of oral masitinib in the treatment of patients with amyotrophic lateral sclerosis (ALS).

Being tested
Masitinib (6.0), Riluzole, Placebo, Masitinib (4.5)
Keywords
Amyotrophic Lateral Sclerosis, ALS, Tyrosine kinase inhibitor, Lou Gehrig's disease, Charcot's disease, Motor Neuron disease, MND
Medical subject headings
Amyotrophic Lateral Sclerosis, Motor Neuron Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Masitinib (6.0)Drug

    Also called AB1010

    Masitinib (titration to 6.0 mg/kg/day)

  • RiluzoleDrug

    Also called Rilutek

    Riluzole 50 mg tablet, treatment per os

  • PlaceboDrug

    Also called Placebo Oral Tablet

    treatment per os

  • Masitinib (4.5)Drug

    Also called AB1010

    Masitinib (titration to 4.5 mg/kg/day)

Study groups in NCT03127267
Study groupRoleWhat they receive
Masitinib (4.5) & RiluzoleExperimentalParticipants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control. Masitinib will be administered as an add-on to riluzole at 50 mg b.i.d
Receives: Drug: Riluzole; Drug: Masitinib (4.5)
Masitinib (6.0) & RiluzoleExperimentalParticipants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control. Masitinib will be administered as an add-on to riluzole at 50 mg b.i.d.
Receives: Drug: Masitinib (6.0); Drug: Riluzole
Placebo & RiluzolePlacebo comparatorParticipants receive a matched dose placebo, given orally twice daily, in combination with riluzole at 50 mg b.i.d.
Receives: Drug: Riluzole; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. ALSFRS-R

    Change in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised.

    Measured: 48 weeks

Secondary outcomes (5)

  1. ALSAQ-40

    Change in ALS quality of life patient questionnaire (ALSAQ-40)

    Measured: 48 weeks

  2. PFS

    Progression free survival (PFS) is defined as the time from randomization to progression (decline of more than 9 points in ALSFRS-R score from baseline) or death

    Measured: From day of randomization to disease progression or death, assessed for a maximum of 36 months

  3. FVC

    Change in Forced Vital Capacity (FVC)

    Measured: 48 weeks

  4. HHD

    Change in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD)

    Measured: 48 weeks

  5. Change in the Combined Assessment of Function and Survival (CAFS) score from baseline to week 48

    CAFS ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. Each patient's outcome is compared to every other patient's outcome, assigned a score, and the summed scores are ranked. The mean rank score for each treatment group can then be calculated. A higher mean CAFS score indicates a better group outcome.

    Measured: 48 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Apr 2017
  2. Study start2 Feb 2021
  3. Primary completionDec 2027 (estimated)
  4. Study completionDec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
495 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03127267
Sponsor's protocol ID
AB19001
Status last verified
Sep 2025
First submitted
13 Apr 2017
Last updated
12 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Latham BD, Oskin DS, Crouch RD, Vergne MJ, Jackson KD. Cytochromes P450 2C8 and 3A Catalyze the Metabolic Activation of the Tyrosine Kinase Inhibitor Masitinib. Chem Res Toxicol. 2022 Sep 19;35(9):1467-1481. doi: 10.1021/acs.chemrestox.2c00057. Epub 2022 Sep 1.

    PubMed 36048877

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Masitinib (6.0), Riluzole and Masitinib (4.5) in people with Amyotrophic Lateral Sclerosis, run by AB Science at 56 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 12 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 56 sites in France, Italy, United States, Spain, Greece and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Clinical Study Coordinator (+33(0)147200014, clinical@ab-science.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

ALSFRS-R (time frame: 48 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03127267, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Sep 2025.

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